Identifying weaknesses in a specific type of childhood cancer
Nominating vulnerabilities in fusion oncoprotein-driven rhabdomyosarcoma
This study is looking at a tough type of cancer called rhabdomyosarcoma that affects kids and young adults, and it aims to find new ways to treat it by understanding the genetic changes caused by a specific protein that drives the disease.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Duke University NIH-funded |
| Lab location | 1 site (Durham, United States) |
| Project ID | NIH-11200239 on NIH RePORTER |
What this research studies
This research investigates rhabdomyosarcoma (RMS), a cancer affecting children and young adults, particularly focusing on the high-risk form driven by the PAX3-FOXO1 fusion protein. The project aims to uncover genetic and epigenetic changes that could be targeted for therapy, using advanced DNA and RNA sequencing techniques. By developing experimental models that can manipulate the expression of the fusion protein, the researchers hope to identify potential therapeutic vulnerabilities that could lead to more effective treatments. This work addresses a critical gap in current treatment options for patients with fusion-positive RMS.
Who could benefit from this research
Good fit: Ideal candidates for this research are children and young adults diagnosed with fusion-positive rhabdomyosarcoma.
Not a fit: Patients with other types of rhabdomyosarcoma or those whose cancer is not driven by the PAX3-FOXO1 fusion protein may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new targeted therapies for children and young adults with high-risk rhabdomyosarcoma, improving their chances of long-term survival.
How similar studies have performed: While research on rhabdomyosarcoma has been ongoing, this specific approach targeting the PAX3-FOXO1 fusion protein represents a novel effort to identify actionable therapeutic targets.
Where this research is happening
Durham, United States
- Duke University — Durham, United States (Active)
Researchers
- Principal investigator: Skapek, Stephen X — Duke University
- Study coordinator: Skapek, Stephen X
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.